ATE224705T1 - Zusammensetzungen mit kationischen amphiphilen und colipiden zur intrazellulären verabreichung therapeutischer moleküle - Google Patents

Zusammensetzungen mit kationischen amphiphilen und colipiden zur intrazellulären verabreichung therapeutischer moleküle

Info

Publication number
ATE224705T1
ATE224705T1 AT97929716T AT97929716T ATE224705T1 AT E224705 T1 ATE224705 T1 AT E224705T1 AT 97929716 T AT97929716 T AT 97929716T AT 97929716 T AT97929716 T AT 97929716T AT E224705 T1 ATE224705 T1 AT E224705T1
Authority
AT
Austria
Prior art keywords
colipides
amphiphils
compositions containing
therapeutic molecules
containing cationic
Prior art date
Application number
AT97929716T
Other languages
German (de)
English (en)
Inventor
Allen J Fasbender
Michael J Welsh
Craig S Siegel
Edward R Lee
Chau-Dung Chang
John Marshall
Seng H Cheng
David J Harris
Simon J Eastman
Shirley C Hubbard
Mathieu B Lane
Eric A Rowe
Ronald K Scheule
Nelson S Yew
Original Assignee
Genzyme Corp
Univ Iowa Res Found
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genzyme Corp, Univ Iowa Res Found filed Critical Genzyme Corp
Application granted granted Critical
Publication of ATE224705T1 publication Critical patent/ATE224705T1/de

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Liposomes
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712Cystic fibrosis

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Pulmonology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Dispersion Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
AT97929716T 1996-06-03 1997-05-30 Zusammensetzungen mit kationischen amphiphilen und colipiden zur intrazellulären verabreichung therapeutischer moleküle ATE224705T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US08/657,238 US5935936A (en) 1996-06-03 1996-06-03 Compositions comprising cationic amphiphiles and co-lipids for intracellular delivery of therapeutic molecules
PCT/US1997/009142 WO1997046223A1 (fr) 1996-06-03 1997-05-30 Compositions comprenant des amphiphiles cationiques et des co-lipides et facilitant l'administration par voie intracellulaire de molecules therapeutiques

Publications (1)

Publication Number Publication Date
ATE224705T1 true ATE224705T1 (de) 2002-10-15

Family

ID=24636394

Family Applications (1)

Application Number Title Priority Date Filing Date
AT97929716T ATE224705T1 (de) 1996-06-03 1997-05-30 Zusammensetzungen mit kationischen amphiphilen und colipiden zur intrazellulären verabreichung therapeutischer moleküle

Country Status (8)

Country Link
US (1) US5935936A (fr)
EP (1) EP0845981B1 (fr)
JP (1) JPH11511757A (fr)
AT (1) ATE224705T1 (fr)
CA (1) CA2228444A1 (fr)
DE (1) DE69715786T2 (fr)
ES (1) ES2187791T3 (fr)
WO (1) WO1997046223A1 (fr)

Families Citing this family (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5674908A (en) 1993-12-20 1997-10-07 Life Technologies, Inc. Highly packed polycationic ammonium, sulfonium and phosphonium lipids
US6989434B1 (en) * 1994-02-11 2006-01-24 Invitrogen Corporation Reagents for intracellular delivery of macromolecules
US5837533A (en) * 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
AU6949098A (en) * 1997-04-04 1998-10-30 Valentis, Inc. Improved methods of delivery using cationic lipids and helper lipids
US6235310B1 (en) 1997-04-04 2001-05-22 Valentis, Inc. Methods of delivery using cationic lipids and helper lipids
EP1027033B1 (fr) * 1997-05-14 2009-07-22 The University Of British Columbia Encapsulation hautement éfficace d'acides nucléiques dans des vésicules lipidiques
US6835395B1 (en) 1997-05-14 2004-12-28 The University Of British Columbia Composition containing small multilamellar oligodeoxynucleotide-containing lipid vesicles
EP1129064B1 (fr) 1998-11-12 2008-01-09 Invitrogen Corporation Reactifs de transfection
US6521431B1 (en) 1999-06-22 2003-02-18 Access Pharmaceuticals, Inc. Biodegradable cross-linkers having a polyacid connected to reactive groups for cross-linking polymer filaments
IL131074A0 (en) 1999-07-23 2001-03-19 Polygene Ltd A biodegradable polycation composition for delivery of an anionic macromolecule
DE60038220T2 (de) * 1999-08-27 2009-03-12 Novartis Vaccines and Diagnostics, Inc., Emeryville Chimerische antisense-oligonukleotide und zelltransfektions-zusammensetzungen davon
US6677445B1 (en) * 1999-08-27 2004-01-13 Chiron Corporation Chimeric antisense oligonucleotides and cell transfecting formulations thereof
EP2336169A1 (fr) * 2000-04-21 2011-06-22 New England Medical Center Hospital Agonistes et antagonistes du récepteur couple à la protine g (GPCR) et procédés d'activation et d'inhibition de GPCR au moyen de ces angonistes et antagonistes
US7696168B2 (en) * 2000-04-21 2010-04-13 Tufts Medical Center, Inc. G protein coupled receptor agonists and antagonists and methods of activating and inhibiting G protein coupled receptors using the same
DE10026698A1 (de) 2000-05-30 2001-12-06 Basf Ag Selbstemulgierende Wirkstoffformulierung und Verwendung dieser Formulierung
WO2001097829A2 (fr) * 2000-06-19 2001-12-27 Genzyme Corporation Traitement de substitution enzymatique, therapie genique et therapie par petites molecules pour maladies liees au stockage lysosomal
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
JP2005502309A (ja) * 2000-12-23 2005-01-27 カイロン コーポレイション オリゴヌクレオチドトランスフェクションスクリーニング方法
IL140844A0 (en) * 2001-01-10 2002-02-10 Polygene Ltd Cationic polysaccharide compositions
US20030096414A1 (en) 2001-03-27 2003-05-22 Invitrogen Corporation Culture medium for cell growth and transfection
WO2002100317A2 (fr) * 2001-05-25 2002-12-19 The Trustees Of The University Of Pennsylvania Particules a destination ciblee et procedes d'utilisation
WO2003035669A1 (fr) * 2001-10-22 2003-05-01 Sterrenbeld Biotechnologie North America, Inc. Lipides cationiques amphiphiles derives du cholesterol
AU2002247919A1 (en) * 2002-03-26 2003-10-08 Council Of Scientific And Industrial Research Cationic amphiphiles for intracellular delivery of therapeutic molecules its composition, process and use thereof
US20040180438A1 (en) * 2002-04-26 2004-09-16 Pachuk Catherine J. Methods and compositions for silencing genes without inducing toxicity
WO2003093449A2 (fr) * 2002-05-06 2003-11-13 Nucleonics, Inc. Procedes d'administration d'acides nucleiques
US7183263B2 (en) 2002-07-17 2007-02-27 University Of Utah Research Foundation Linear polyethylenimine-sterol conjugates for gene delivery
AU2003274906A1 (en) * 2002-07-31 2004-02-16 Nucleonics, Inc. Double stranded rna structures and constructs, and methods for generating and using the same
EP1578193A4 (fr) * 2002-12-23 2011-06-15 Vical Inc Procede de lyophilisation de complexes d'acide nucleique/copolymere sequence/tensioactif cationique
US8025899B2 (en) 2003-08-28 2011-09-27 Abbott Laboratories Solid pharmaceutical dosage form
US8377952B2 (en) 2003-08-28 2013-02-19 Abbott Laboratories Solid pharmaceutical dosage formulation
JP4944032B2 (ja) 2004-09-13 2012-05-30 ジェンザイム・コーポレーション 多量体構築物
BRPI0517058A (pt) * 2004-11-04 2008-09-30 New England Medical Center Inc agonistas e antagonistas do receptor acoplado à proteìna g e métodos de uso
DK2494993T3 (en) 2007-05-04 2018-11-12 Marina Biotech Inc Amino acid lipids and uses thereof
SI2889043T1 (sl) 2008-12-16 2019-08-30 Genzyme Corporation Sintetične vmesne spojine za pripravo konjugatov oligosaharid-protein
NZ700688A (en) 2009-12-01 2016-02-26 Shire Human Genetic Therapies Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
JP6184945B2 (ja) 2011-06-08 2017-08-23 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド mRNA送達のための脂質ナノ粒子組成物および方法
EP3536787A1 (fr) 2012-06-08 2019-09-11 Translate Bio, Inc. Polynucléotides résistant aux nucléases et leurs utilisations
EA037922B1 (ru) 2013-03-14 2021-06-07 Шир Хьюман Дженетик Терапис, Инк. мРНК CFTR И КОМПОЗИЦИИ ДЛЯ ЛЕЧЕНИЯ МУКОВИСЦИДОЗА У МЛЕКОПИТАЮЩЕГО
EP2970955B1 (fr) 2013-03-14 2018-11-14 Translate Bio, Inc. Procédés de purification d'arn messager
ES2954366T3 (es) 2013-10-22 2023-11-21 Translate Bio Inc Terapia de ácido ribonucleico mensajero para la deficiencia de argininosuccinato sintetasa
CA2928186A1 (fr) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Therapie a l'arnm pour la phenylcetonurie
JPWO2015093588A1 (ja) * 2013-12-21 2017-03-23 Ktnバイオテック株式会社 抗癌剤
ES2750661T3 (es) 2014-04-25 2020-03-26 Translate Bio Inc Métodos para la purificación de ARN mensajero
WO2016011203A1 (fr) 2014-07-15 2016-01-21 Life Technologies Corporation Compositions comprenant des agrégats lipidiques et procédés d'administration efficace de molécules dans des cellules
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
IL270631B2 (en) 2017-05-16 2024-03-01 Translate Bio Inc Treatment of cystic fibrosis through the administration of mRNA with an optimal codon encoding ctfr
CN112930396B (zh) 2018-08-24 2024-05-24 川斯勒佰尔公司 用于纯化信使rna的方法

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4394448A (en) * 1978-02-24 1983-07-19 Szoka Jr Francis C Method of inserting DNA into living cells
US4544545A (en) * 1983-06-20 1985-10-01 Trustees University Of Massachusetts Liposomes containing modified cholesterol for organ targeting
JPS617292A (ja) * 1984-06-20 1986-01-13 Japan Atom Energy Res Inst 制癌剤を結合させたエストラサイト系化合物を製造する方法
US4897355A (en) * 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4971803A (en) * 1985-04-08 1990-11-20 California Institute Of Technology Lamellar vesicles formed of cholesterol derivatives
JPH02500360A (ja) * 1986-07-28 1990-02-08 リポソーム テクノロジー,インコーポレイテッド 粘膜組織上での高い保持力を有するリポソーム
US5043164A (en) * 1989-01-17 1991-08-27 The University Of Tennessee Research Corporation Blood-stable, cholesterol-free liposomes
FR2645866B1 (fr) * 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US4958013A (en) * 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5004737A (en) * 1989-09-22 1991-04-02 Pacific Chemical Co., Ltd. Quaternary ammonium-substituted sterol derivatives
CA2037478A1 (fr) * 1990-03-05 1991-09-06 Richard Gregory Methodes de diagnostic et de traitement faisant appel a un regulateur membranaire de la fibrose kystique
US5279833A (en) * 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5264618A (en) * 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
EP0490379A3 (fr) * 1990-12-13 1992-06-24 BERLIN-CHEMIE Aktiengesellschaft Dérivés de diamino-acides et compositions pharmaceutiques
US5283185A (en) * 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5334761A (en) * 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
US5705655A (en) * 1992-12-17 1998-01-06 Megabios Corporation Amphiphilic nitrogen containing imidazolinium derivative compounds and uses
DE69412596T2 (de) * 1993-03-10 1999-03-04 Magainin Pharma Steroidderivate, pharmazeutische zusammensetzungen die sie enthalten und ihre verwendung als antibiotika oder desinfektionsmittel
US5578475A (en) * 1993-07-12 1996-11-26 Life Technologies, Inc. Composition and methods for transfecting eukaryotic cells
NZ276975A (en) * 1993-11-24 1998-04-27 Megabios Corp Guanidine based amphiphiles and their use in liposomes for delivering genetic material intracellularly
CA2176714A1 (fr) * 1993-11-24 1995-06-01 Timothy D. Heath Derives amphiphiles de piperazine
JPH09508099A (ja) * 1993-12-20 1997-08-19 ユニヴェルシテ リブル ドゥ ブリュッセル 細胞中に少なくとも一つの分子を導入できる化合物
AU1919295A (en) * 1994-02-14 1995-08-29 Indiana University Foundation Aminophospholipid compositions and uses thereof
WO1995024222A1 (fr) * 1994-03-07 1995-09-14 University Of Medicine & Dentistry Of New Jersey Produits de conjugaison polymeres polycationiques cycliques-oligonucleotides et procedes de preparation
US5670347A (en) * 1994-05-11 1997-09-23 Amba Biosciences Llc Peptide-mediated gene transfer
US5908635A (en) * 1994-08-05 1999-06-01 The United States Of America As Represented By The Department Of Health And Human Services Method for the liposomal delivery of nucleic acids
US5837533A (en) * 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
US6071890A (en) * 1994-12-09 2000-06-06 Genzyme Corporation Organ-specific targeting of cationic amphiphile/DNA complexes for gene therapy
WO1996020208A2 (fr) * 1994-12-28 1996-07-04 Max-Delbrück-Centrum für Molekulare Medizin Nouveau derive de cholesterol pour le transfert genique liposomal
FR2744453B1 (fr) * 1996-02-05 1998-07-10 Transgene Sa Composes lipopolyamines, compositions pharmaceutiques en comprenant, vecteurs de transfert d'acide nucleique et procede de preparation

Also Published As

Publication number Publication date
JPH11511757A (ja) 1999-10-12
EP0845981A1 (fr) 1998-06-10
EP0845981B1 (fr) 2002-09-25
US5935936A (en) 1999-08-10
DE69715786T2 (de) 2003-05-28
DE69715786D1 (de) 2002-10-31
WO1997046223A1 (fr) 1997-12-11
CA2228444A1 (fr) 1997-12-11
ES2187791T3 (es) 2003-06-16

Similar Documents

Publication Publication Date Title
ATE224705T1 (de) Zusammensetzungen mit kationischen amphiphilen und colipiden zur intrazellulären verabreichung therapeutischer moleküle
ATE203019T1 (de) Neue (r)-5-carbamoyl-8-fluor-3-n,n- disubstituierte-amino-3,4-dihydro-2h-1- benzopyrane
BG104315A (en) Prostaglandine agonists and their application for the treatment of osteal diseases
DE69627690D1 (de) Liposomale formulierungen von mitoxantron
ES2168650T3 (es) Compuestos y metodos moduladores de receptores de androgenos.
DE69729786D1 (de) Hochkonzentrierte, lyophilisierte und flüssige faktor-ix formulierungen
ME00513B (fr) Azabicycloalcanes modulateurs de ccr5
YU40701A (sh) Piperidini kao modulatori ccr5
ATE210988T1 (de) Zytotoxischer aminozucker und verwandte zuckerderivate von indolopyrrolocarbazolen
NO963850L (no) Heterosykliske forbindelser, deres fremstilling og anvendelse
ATE223399T1 (de) Quinazolin-4-on ampa antagonisten
ATE188873T1 (de) Tris-platin-komplexe
ATE297396T1 (de) Neue atropisomere von 2,3-disubstituierten-(5,6)- heteroarylkondensierten-pyrimidin-4-onen
DE59608150D1 (de) Transdermales therapeutisches system (tts) zur verabreichung von testosteron
AU7525398A (en) Inhibitors for urokinase receptor
ES2161913T3 (es) Complejos cationicos trinucleares de platino que tienen una actividad antitumoral y composiciones farmaceuticas que los contienen.
NO990322L (no) Farmas°ytisk blanding inneholdende en 5HT2c-antagonist og en D2-antagonist

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties